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A Rare Approach TO THERAPEUTICS J.P . MORGAN HEALTHCARE CONFERENCE JANUARY 2026 Nasdaq: ZVRA Adult living with Niemann-Pick disease type C CRC-ZEV-26-0006
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Forward Looking Statements 2 Presentation may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements relate to future events or our future financial performance. We generally identify forward-looking statements by terminology such as “ m a y,” “will, ” “would, ” “should, ” “e x p e c t s ,” “plans, ” “anticipates, ” “could, ” “intends, ” “target, ” “projects, ” “contemplates, ” “believes, ” “estimates, ” “predicts, ” “assume, ” “intend, ” “potential, ” “continue” or other similar words or the negative of these terms. We have based these forward-looking statements largely on our current expectations about future events and financial trends that we believe may affect our business, financial condition and results of operations. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements include statements regarding the promise and potential impact of our preclinical or clinical trial data, including without limitation the timing and results of any clinical trials or readouts, our anticipated financial performance, our industry, our intellectual property, business strategy, plans, goals and expectations concerning our market position, future operations, the timing or results of any investigational New Drug Applications (NDA) and NDA submissions, the timing or results of any Marketing Authorization Application (MAA) and MAA submissions, communications with the U.S. Food and Drug Administration (FDA) and/or the European Medicines Agency (EMA), the potential uses or benefits of MIPLYFFA, KP1077, SDX, celiprolol or any other product candidates for any specific disease indication or at any dosage, the potential benefits of any of Zevra’s product candidates, the success or timing of the launch or commercialization of any products or related sales milestones, and our strategic and product development objectives. These forward-looking statements are based on information currently available to Zevra and its current plans or expectations and are subject to a number of known and unknown uncertainties, risks and other important factors - including those discussed under the caption “Risk Factors” in Zevra’s Annual Report on Form 10-K for the year ended December 31, 2024, filed on March 12, 2025, Quarterly Report on Form 10-Q for the three and nine months ended September 30, 2025, filed on November 5, 2025, and in our other filings with the SEC – and could cause actual results, performance, or achievements to differ materially from those indicated by the forward-looking statements made herein. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to this presentation. This presentation also may contain estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk.
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To become a leading rare disease therapeutics company that is driven by patient insights and innovation to make a transformational impact on the people we serve We are a Purpose Driven Company 3 We redefine what is possible in bringing life-changing therapies to people living with rare diseases Patient Centricity prioritizing patient needs Accountability delivering on commitments consistently Integrity always doing what is right Innovation transforming possibilities into reality Courage acting with strength and conviction OUR VISION OUR MISSION OUR VALUES
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Late-stage clinical development assets Unique Opportunity to Impact People Living with Rare Diseases 4i. 9-month period through Sept 30, 2025; ii. Cash, cash equivalents and investments as of Sep. 30, 2025 Commercial-stage rare disease products Existing infrastructure can be leveraged for future growth Exited Q3 25 with a strong cash position: $230.4Mii Growth mode; operating runway to execute MAA under review by EMA Multiple revenue sources generated strong net revenue of $72.3M in 9-monthsi Adult living with NPC
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PHASE 1 PHASE 2 PHASE 3 NDA/MAAi APPROVED STATUS & IP U.S Commercial FDA Approval: Sep 20, 2024 Protection through 2031 via ODE ii FDA Approval: Dec 22, 2022 IP through 2036 Partnered Receiving royalties and milestones on net sales iii IP through 2037 EU regulatory Late-stage Development MAA under review by EMA Submitted Jul 28, 2025 Ph. 3 trial ongoing IP potential through 2038 Ph. 3 trial ready IP potential through 2038 Ph. 3 trial potential Diversified Portfolio Delivering Value for Patients 5 Certain products may be subject to royalty obligations, details and required disclosures are available inour SEC filings or on our website: www.zevra.com. i. New Drug Application\Marketing Authorization Application; ii. Orphan Drug Exclusivity; iii. Zevra partnered asset. The safety and efficacy of product candidates have not been established. There is no guarantee that these products will receive health authority approval or become commercially available for the uses being investigated. MIPLYFFA® arimoclomol Niemann-Pick Disease Type C (NPC) OLPRUVA® sodium phenylbutyrate for oral suspension Urea Cycle Disorders (UCD) AZSTARYS® serdexmethylphenidate and dexmethylphenidate Attention Deficit Hyperactivity Disorder (ADHD) Arimoclomol Niemann-Pick Disease Type C (NPC) Celiprolol Vascular Ehlers-Danlos Syndrome (VEDS) KP1077 Idiopathic Hypersomnia (IH) KP1077 Narcolepsy
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Resources Prioritized Across Four Pillars to Achieve our Vision Fulfilling Our Mission to Bring Life-Changing Therapies to People Living with Rare Disease 6 Focus Execute Innovate Marketed Therapies Pipeline and Innovation Talent and Culture Corporate Foundation Growth mode for MIPLYFFA® to deliver meaningful benefit to patients Synergistic growth opportunities Experienced team with rare disease expertise Strong balance sheet with financial discipline
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Execution in 2025 Paved the Way for Multiple Growth Drivers in 2026 7 Marketed Products Pipeline and Innovation Talent and Culture Corporate Foundation • Adoption achieved in ~40% of diagnosed patients within first year of launch Significant Growth Opportunities Becoming “Partner of Choice” for Rare Disease Products Significant Revenue Generation & $150M PRV Monetization Established MIPLYFFA as Foundational Treatment for NPC • Arimoclomol MAA under review by EMA • 92 expanded access patients in EU/UK • Broadening expanded access programs (EAP) to territories outside EU/UK • Pivotal Phase 3 trial underway for celiprolol • Demonstrating leadership through advocacy, community engagement, and patient and caregiver support services • Established presence in rare disease biotech hub • Strong balance sheet • Demonstrated operational discipline • Bolstered financial flexibility
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Marketed Therapies Niemann-Pick disease type C (NPC) is an ultra-rare genetic disorder that leads to premature mortality. Adult living with NPC Adult living with NPC For full prescribing information, visit MIPLYFFA.com
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Signs and Symptoms of NPC: NPC is a Devastating and Fatal Lysosomal Storage Disorder 9 NPC gene mutations produce abnormal, absent, or non-functional NPC proteins1 Progressive lipid build up leads to cell death and ultimately organ dysfunction in the spleen, liver, and brain Cholesterol Buildup Leads to Cell Death Age of Onset Split 50/50 (children/adults) Rate of Progression Presentation of Symptoms Heterogenous Presentation Cognition Speech Ambulation Swallow Fine Motor Skills Visceral
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Multiple Trials Demonstrate MIPLYFFA’s Impact on NPC Progression Proven effectiveness using the NPC Clinical Severity Scale in combination with miglustat i,2, 3 10iMeasured by the R4DNPCCSS score: ambulation, speech, swallow, and fine motor skills. 80% of patients were taking migulstat. R4DNPCCSS over 48 months OLE with MIPLYFFA + miglustat Months since OLE baseline ~80% Of patients who participated in the Phase 2/3 clinical trial took miglustat2 Changing the Therapeutic Landscape of NPC Durable effect for 5+ Years2,3 Halts Disease Progression at 12 months2,3 Improvement at first evaluation Week 122,3 For full prescribing information, visit MIPLYFFA.com
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Establishing MIPLYFFA as Foundational Treatment for NPC 11 Demonstrating Clinical Differentiation Driving Patient Access Increasing Identification of Patients • Unique mechanism of action improves cholesterol trafficking through improved lysosomal function and upregulation of genes belonging to the CLEAR networki,5 • Strength of clinical data • Magnitude of safety database • Robust and expanding body of evidence • Payor coverage at 66% of covered lives as of Q3 25 • Patients gaining coverage through Medical Exception Pathways, if not on formulary • Driving conversion of Patient Enrollments and High Adherence Rates • 137 patient enrollments; 8 in Q3 25 ii • Programs implemented to find undiagnosed patients yielding encouraging results • Expanded genetic Dx collaboration yielding new patient identification • Initiated AI predictive model to find potential patients Independent market research suggests MIPLYFFA is the preferred NPC therapy most trusted by clinicians and shown to improve balance, swallowing, cognition, speech, and reduce falls. i. Coordinated Lysosomal Expression and Regulation (CLEAR); ii. As of Q3 25 For full prescribing information, visit MIPLYFFA.com
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Capturing the Next Wave of Growth for MIPLYFFA ~900 individuals in the U.S. live with NPC of which 300-350 are diagnosed or treated4 • Continuing to drive penetration amongst currently Dx patients • Finding Dx but not treated patients • Helping get new patients Dx & treated earlier • MAA under review by EMA • Submission includes most expansive data set available in NPC • Expanding EAP to support more patients 12 U.S. Prevalence ~1,100 individuals living with NPC in Europe5 Diagnosis rates estimated to be higher than the U.S. Geographic expansion to broaden access outside the U.S. and Europe • Supporting a greater number of patients • Furthers mission to provide access • Building distributor network in select territories Expanded AccessEU Prevalence
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Pipeline and Innovation Vascular Ehlers-Danlos Syndrome (VEDS) is a severe autosomal, dominant, genetic, connective tissue disorder.
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Leads to defect in type III procollagen in vessel walls and hollow organs Characterized by arterial aneurysms and hollow organ ruptures6 VEDS is a Life-threatening Connective Tissue Disorder with No Approved Treatments 14 ~7,500 individuals in the U.S. diagnosed and live with VEDS7 95% of diagnoses are genetically confirmed (COL3A1)7, 8 ~25% of patients experience an event before the age of 207 ~90% of patients experience an event by the age of 407 25% 90% Median survival age 51 YEARS Arterial rupture being the most common cause of sudden death7 VEDS is the most severe Ehlers-Danlos syndrome subtype6 Inherited connective tissue disorder caused by COL3A1 gene mutations
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Celiprolol is a Potential Treatment of Patients with COL3A1+ VEDS 15 • Celiprolol addresses VEDS by reducing mechanical stress on collagen fibers within arterial wall9 • New chemical entity in the U.S., Orphan drug & breakthrough therapy designation • Selective adrenergic modulator (SAM) • Randomized & real-world data across BBEST and two long-term European cohorts • ~5% annual major vascular event rate on celiprolol vs ~12% untreated • Improved survival observed in treated patients • Decentralized pivotal trial (in-home & virtual) • Special Protocol Assessment (SPA) • Designed to confirm clinical benefit seen in prior studies • 44 of 150 patients enrolled as of Q3 25 • Interim analysis at 28 events; final at 46 events Celiprolol is the primary treatment for VEDS patients in several European countries13 Consistent Reduction in Major Vascular Events Across Previously Completed Studies 10,11,12 Phase 3 DiSCOVER Trial Ongoing
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Strong Corporate Foundation Demonstrating Financial Discipline • Available cash, cash equivalents and investments of $230.4M BALANCE SHEET AS OF SEPT 30, 2025:
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Financial Position is a Source of Strength 17 Q3 2025 Income Statement Details: • Available cash, cash equivalents and investments of $230.4M – Debt of $61.3M Balance Sheet as of Sept 30, 2025: Net revenue of $26.1M for Q3 MIPLYFFA: $22.4M AZSTARYS license agreement: $1.2M French EAP net reimbursements: $2.4M OLPRUVA: $100K Q3 2025 net loss of $(0.5)M, or $(0.01) per basic and diluted share • Includes non-cash warrant fair value adjustment of $5.5M, non-cash stock-based compensation expense of $2.8M, and non-cash intangible asset amortization of $0.3M
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Growth Drivers to Maximize the Opportunity in 2026 and Beyond 18 Commercial Execution to Further Unlock NPC Opportunity in the U.S. • Continue successful execution of U.S. launch to drive access to prevalent patient population • Genetic testing collaborations to find undiagnosed patients • Publication strategy to drive clinical differentiation and continuing education Possible Patent Term Extension for MIPLYFFA • Magnitude of each additional year meaningful • Additional IP filed with USPTO in 2024 and 2025 MAA Submitted; Potential for EMA Approval of Arimoclomol • Continue to drive EU experience and KOL network through EAP • Go to market strategy in Europe Geographic expansion to broaden access to patients beyond U.S. and Europe
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A Rare Approach to Therapeutics Nasdaq: ZVRA Children living with NPC
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Sources 1. Geberhiwot T, et al. Orphanet J Rare Dis. 2018 Apr 6;13(1):50. 2. MIPLYFFA Full Prescribing Information. Celebration, FL, US, Zevra Therapeutics Inc.; 09/2024. 3. Mengel et al., Molecular Genetics and Metabolism Volume 145, Issue 4, August 2025, 109189. https://doi.org/10.1016/j.ymgme.2025.109189 4. Burton et.al., Molecular Genetics and Metabolism Volume 134, Issues 1–2, September–October 2021, Pages 182-187 5. Patterson M. Niemann-Pick Disease Type C. 2000 Jan 26 [Updated 2020 Dec 10]. In: Adam MP , Feldman J, Mirzaa GM, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2025. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1296/ 6. FightvEds.org 7. Pepin M, et al. Genet Med. 2014 Dec;16(12):881-8 8. Leistritz, D., Pepin, M., Schwarze, U.et al. COL3A1 haploinsufficiency results in a variety of Ehlers-Danlos syndrome type IV with delayed onset of complications and longer life expectancy. Genet Med 13, 717–722 (2011). https://doi.org/10.1097/GIM.0b013e3182180c89 9. Nawarskas J, et al. Cardiology in Review: Celiprolol: a Unique Selectve Adrenoceptor Modulator. Sept/Oct 2017, Volume 25, Number 5 247-253 10. Ong et al., Lancet 2010; 376: 1476-1484 11. Frank et al., J Am Coll Cardiol. 2019 Apr 23;73(15):1948-1957 12. Baderkhan et al., Eur J Vasc Endovasc Surg (2021) 61, 326-331 13.FightvEds.org 20